FDA meeting on Thursday to focus on improving patient engagement
Virtual public meeting aims to gain patient perspective on rare diseases
The U.S. Food and Drug Administration (FDA), in collaboration with Duke University’s Duke-Margolis Center for Health Policy, will hold a virtual public meeting on Thursday, Dec. 14 with an aim toward ultimately improving patient community engagement in the development of rare disease treatments — including ones for aromatic l-amino acid decarboxylase (AADC) deficiency.
The online workshop, titled “Advancing the Development of Therapeutics Through Rare Disease Patient Community Engagement,” will be held from 12 p.m. to 5 p.m. ET. Anyone interested in participating may register via the Duke-Margolis Center website.
“This public meeting will discuss approaches and opportunities for engaging patients, patient groups, [and] rare disease or condition experts … during the drug development process for rare diseases,” the FDA stated in a press release announcing the event.
Required by the Food and Drug Omnibus Reform Act of 2022, the meeting “will focus on how to best understand patients’ experiences living with a rare disease and how to incorporate those experiences and priorities throughout the drug development process,” the release stated.
Specifically, its aim is to gain patient perspectives on the burden of their condition and any existing treatment options, and to learn how patients’ current health status, and the risk of disease progression, affects their willingness to accept any treatment side effects.
NORD also hosting RDAC Stakeholders Meeting on Thursday
Upon registering for the meeting, attendees are encouraged to submit questions, which will be incorporated into moderated discussions during the event. Questions also may be submitted live through the webinar’s question-and-answer function.
In addition, stakeholder comments regarding the virtual meeting may be submitted to the FDA docket FDA-2023-N-4718, which will remain open until Feb. 12.
According to the National Organization for Rare Disorders (NORD), about 1 in 10 U.S. residents — about 30 million people in all — live with a rare disorder, such as AADC deficiency. Of the more than 7,000 known rare conditions, fewer than 5% have an approved treatment.
“Traditional clinical trials are challenging to conduct for therapies targeting these small populations due to a limited understanding of disease natural history, difficulty powering traditional clinical trials given small population size and disease heterogeneity [variability], and challenges with identifying and recruiting participants. Together, these factors significantly impact the rare disease drug development space,” the Duke-Margolis Center states in the event’s page.
Understanding the experiences and priorities of patients and working closely with rare disease or condition experts, and experts on small population studies can be important for informing and supporting the drug development process for products intended for patients with rare diseases.
The Center notes that developing new treatments is “critical,” particularly given that nearly half of rare diseases affect children. Further, rare diseases often are progressive, and many are life-threatening.
“Given these challenges, understanding the experiences and priorities of patients and working closely with rare disease or condition experts, and experts on small population studies can be important for informing and supporting the drug development process for products intended for patients with rare diseases,” the event’s page states.
Last year, the gene therapy Upstaza (eladocagene exuparvovec) was approved for AADC deficiency patients, 18 months and older, in the European Union and the U.K.
PTC Therapeutics, the treatment’s developer, is slated to meet with the FDA this month to review the data it plans to include in a regulatory application seeking Upstaza’s approval in the U.S.
Also on Dec. 14, NORD will host its fourth annual Rare Disease Advisory Council (RDAC) Stakeholders Meeting from 1 p.m. to 2:30 p.m. ET. The virtual event will involve NORD, RDAC members, and rare disease advocates nationwide.
Anyone interested in learning more about RDACs — advisory bodies that seek to give the rare disease community a stronger voice in state governments — and statewide advocacy of rare disorders, are encouraged to register for that meeting.
The meeting will provide updates from a number of the newly formed RDACs and introduce ways for attendees to participate in their state’s RDAC in the run up to the establishment of 2024 legislative calendars.
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